A day after announcing a late-stage trial of its lead drug for a rare disease of the kidneys and heart, Alnylam Pharmaceuticals said on Monday that the experimental drug could get faster-than-normal approval in the U.S.
Alnylam, in Cambridge, is working on several drugs for rare diseases that work by turning off specific genes. It’s most advanced one is an intravenous drug called patisiran, intended for transthyretin (TTR)-familial amyloid polyneuropathy, or ATTR, which affects about 40,000 people in the U.S. The company also has several others in the works with the goal of having five potential drugs in human trials by the end of 2015.
Over the weekend, the company presented data from a mid-stage trial of the drug involving 29 ATTR patients which showed the drug reduced levels of the main protein involved in the disease by an average of 85 percent. It also detailed plans to begin a late-stage, placebo-controlled study of the drug, which will enroll up to 200 patients, administering the drug every three weeks for a year and a half.
On Monday, the company further announced that patisiran has received fast track designation from the U.S. Food and Drug Administration, which is aimed at getting drugs for serious diseases without any currently available treatments to market faster. The designation affords frequent meetings with the FDA during the development phase and eligibility for accelerated approval.
On Monday, the company’s stock opened up 2.5 percent from Friday’s close to $54.31 in the first few minutes of trading.
In a research note after the data presentation, Leerink Swann analyst Marko Kozul said the data and the design of the Phase 3 trial were both promising, and he raised his price target on the company’s stock from $80 to $82 a share.